Capricor Therapeutics to Present Exosome Platform Updates at 2024 American Association of Extracellular Vesicles Annual Meeting
Abstract Highlights New Preclinical Data on Exosome-Based Delivery of PMOs for the Treatment of DMD-SAN DIEGO, Nov. 12, 2024 ...
precisionmedicineonline7d
Sarepta Stops Duchenne Drug Development; Nets Higher-Than-Expected Elevidys Q3 Sales
SRP-5051 is designed to bind to exon 51 of dystrophin pre-mRNA, resulting in exclusion of this exon during mRNA processing in patients with genetic mutations amenable to exon 51 skipping. With this ...
eLife1mon
Genetic Inactivation of the β1 adrenergic receptor prevents Cerebral Cavernous Malformations in zebrafish
The authors follow up on a prior paper in which they showed that beta1 adrenergic receptors contributed to the pathogenesis of cavernous malformations. In the prior work, they used morpholinos and ...
eLife2y
Clathrin-independent endocytic retrieval of SV proteins mediated by the clathrin adaptor AP-2 at mammalian central synapses
Neurotransmission is based on the exocytic fusion of synaptic vesicles (SVs) followed by endocytic membrane retrieval and the reformation of SVs. Conflicting models have been proposed regarding the ...
ki.se3y
Division of Biomolecular and Cellular Medicine (BCM) at Labmed
Activities at the Division of Biomolecular and Cellular Medicine (BCM) focus on the application of modified biomolecules, cells and cell products as novel therapy agents as well as the identification ...